Gene therapy for epilepsy: An emerging, promising approach for a serious neurological disorder
Abstract Gene therapy is emerging as a groundbreaking strategy for treating epilepsy, offering new hope to patients who do not respond to conventional medications. Despite advancements in anti‐seizure treatments, nearly 30%–40% of individuals with epilepsy continue to experience uncontrolled seizures, highlighting the urgent need for more effective and
Marco Ledri, Merab Kokaia
wiley +1 more source
Rescue of splicing-mediated intron loss maximizes expression in lentiviral vectors containing the human ubiquitin C promoter. [PDF]
Lentiviral vectors almost universally use heterologous internal promoters to express transgenes. One of the most commonly used promoter fragments is a 1.2-kb sequence from the human ubiquitin C (UBC) gene, encompassing the promoter, some enhancers, first
Cooper, Aaron R +3 more
core +1 more source
Cas9‐Embedding Hyperactive TadA8e Confers Efficient and Highly Specific A‐To‐G Base Editing in Rice
ABSTRACT Adenine base editors (ABEs) produce precise A‐to‐G conversion in the genomic target sites without causing double‐strand breaks. However, the hyperactive adenosine deaminase TadA8e raises safety concerns on genome‐wide off‐target edits. We engineered 11 chimeric proteins for ABEs (CP‐ABEs) by embedding hyperactive TadA8e within Cas9 nickase to ...
Jianjian Hu +14 more
wiley +1 more source
Adenosine deaminase (ADA; Ec: 3.5.4.4), 5´- Nucleotidase (5´– NT; Ec: 3.1.3.5), and AMP – amino hydrolase (AMP – deaminase AMPDA; Ec: 3.5.4.6) activities were measured in sera of ovarian cancer patients before surgery, and after chemotherapy.
Baghdad Science Journal
doaj +1 more source
DNA Methylation Episignature as a Novel Diagnostic Tool for Diamond‐Blackfan Anemia Syndrome
Diamond‐Blackfan Anemia Syndrome (DBAS) is a congenital bone marrow failure disorder characterized by defective erythropoiesis and a spectrum of congenital anomalies, including craniofacial malformations, limb abnormalities, and cardiac and renal defects.
Paola Quarello +29 more
wiley +1 more source
SIGNIFICANCE OF ADENOSINE DEAMINASE SERUM CONCENTRATIONS IN THE DIAGNOSIS OF EXTRA-PULMONARY TUBERCULOSIS [PDF]
Extra pulmonary tuberculosis (EPTB) is a growing problem worldwide. Due to the nature of the disease, the diversity of clinical pictures as well as its minor epidemiological importance, the diagnosis is difficult and often late.In addition to standard TB
Stevanovic G, +6 more
doaj
Novel mutation in CECR1 gene associated with deficiency of adenosine deaminase -2 presenting as severe congenital neutropenia [PDF]
Pronamee Borah +4 more
openalex +1 more source
In Vitro and Clinical Evaluation of Potential Interactions of Bemnifosbuvir with Drug Transporters
Abstract Bemnifosbuvir is a novel oral guanosine nucleotide prodrug candidate for the treatment of chronic hepatitis C virus infection. Potential drug–drug interactions (DDIs) of bemnifosbuvir as a substrate or perpetrator with regard to ATP‐binding cassette (ABC) and solute carrier (SLC) transporters were evaluated in vitro and in clinical studies ...
Xiao‐Jian Zhou +9 more
wiley +1 more source
EIF4A3‐Induced Circular RNA circSnd1 Promotes Muscle Atrophy and Muscle Ageing by Stabilizing EEF1A1
ABSTRACT Background Muscle atrophy is a common complication of ageing, and many chronic conditions, lacks defined therapeutic interventions. It is still mostly unknown how circular RNAs contribute to muscle atrophy. Methods circRNA sequencing and quantitative real‐time PCR were performed to detect the changed circRNAs in muscle atrophy models and aged ...
Jin Li +21 more
wiley +1 more source

